[CIS PIDD] [cis-pidd] Bone marrow transplant for X-linked CGD

[Cowan, Mort]

That is certainly an option although we have seen significant rejection of 1 antigen/allele mismatched unrelated donors when using targeted Bu Flu and ( rATG or Alemtuzumab) (Law et al, BBMT 18:1656, 2012).

Mort

Morton J. Cowan, M.D.
Professor of Pediatrics
Chief, Allergy, Immunology, and Blood and Marrow Transplant Division
UCSF Children's Hospital, Room M659
505 Parnassus Ave
San Francisco, CA 94143-1278

Phone: 415-476-2188
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From: Elie Haddad [mailto:elie.haddad at umontreal.ca]
Sent: Friday, October 19, 2012 6:34 AM
To: CIS-PIDD
Subject: Re: [cis-pidd] Bone marrow transplant for X-linked CGD

I do agree with Michael, I would go with the matched (1 A mismatched)  unrelated donor with the conditionning regimen of Tayfun Gungor.
Elie


Elie Haddad, MD, PhD,
Professor of Pediatrics, University of Montreal,
Head, Pediatric Immunology and Rheumatology Division,
CHU Sainte-Justine, 3175 Cote Sainte-Catherine
Montreal, QC, H3T 1C5, Canada
Ph: 1 514 345 4713
fax: 1 514 345 4897
e-mail: elie.haddad at umontreal.ca<mailto:elie.haddad at umontreal.ca>





Le 2012-10-19 à 02:04, Michael Albert a écrit :


If the unrelated donor is a high-resolution typed full match at B, C, DR, DQ and has the one mismatch at A, this would be our donor choice. Haploidentical parent would be next. In any case we would go for submyeloablative Bu with Flu and Alemtuzumab, which has shown excellent results (Güngör et al, abstract at ESID 2012 in Clinical Immunology).
The Alemtuzumab will also be helpful to treat the hyperinflammation/secondary HLH, but this should be best be in remission before transplant, if possible.
Best,
Michael


PD Dr. med. Michael Albert

Oberarzt

Abteilung für Pädiatrische Hämatologie/Onkologie

Leiter der Stammzelltransplantation

Dr. von Haunersches Kinderspital der LMU

Lindwurmstr.4

80337 München

Tel: 089 5160 2785

Fax: 089 5160 4719

Am 19.10.2012 um 05:50 schrieb "Newburger, Peter" <Peter.Newburger at umassmed.edu<mailto:Peter.Newburger at umassmed.edu>>:
I can't comment on the donor/HSCT issues, but would suggest that you evaluate and possibly treat the patient for secondary HLH prior to transplant.
Regards,
Peter Newburger
-------------------------------------------------------------
Peter E. Newburger, MD
Ali and John Pierce Professor of
     Pediatric Hematology/Oncology
Vice Chair for Research
Department of Pediatrics
University of Massachusetts Medical School
55 Lake Avenue North
Worcester, MA 01655
tel. 508 856-4225
fax 508 856-4282
peter.newburger at umassmed.edu<mailto:peter.newburger at umassmed.edu>
-------------------------------------------------------------

________________________________
From: Howard Lederman [hlederm1 at jhmi.edu<mailto:hlederm1 at jhmi.edu>]
Sent: Thursday, October 18, 2012 2:00 PM
To: CIS-PIDD
Subject: [cis-pidd] Bone marrow transplant for X-linked CGD
We have a 4 y/o boy with X-linked CGD who has had a stormy course with a history of multiple infections  and mild inflammatory bowel disease.  He currently has  generalized adenopathy, hepatosplenomegaly and bone marrow granulomas without an identifiable microbial cause.  He has not responded to a variety of antibiotics, but is improving with  corticosteroids. We would like to do a bone marrow transplant.

The available donor options are:


1.       Unrelated  4/6 matched cord blood (match at DRB1)

2.       Haploidentical parent (probably the father, as the mother has a mixed connective tissue disease)

3.       One possible NMDP unrelated donor who is mismatched at A

I am interested in the experience of others with donor choice.  I am also interested in recommendations for full myeloablation  vs. reduced intensity conditioning, particularly in view of the recent publication from Duke (Tewari et al 2012.  Biol Blood Marrow Transplant 18:1368-77).


Howard M. Lederman, M.D., Ph.D.
Professor of Pediatrics, Medicine and Pathology Division of Pediatric Allergy and Immunology Johns Hopkins Hospital - CMSC 1102 600 N. Wolfe Street Baltimore, MD 21287-3923
Phone: 410-955-5883
Fax: 410-955-0229
Email: Hlederm1 at jhmi.edu<mailto:Hlederm1 at jhmi.edu>

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